JERRY LEWIS LABOR DAY TELETHON WILL FEATURE
MAJOR MDA RESEARCH ADVANCES IN 2006-2007

TUCSON, Ariz., Aug. 1, 2007 – Gene therapy, gene modification, enzyme replacement and large-scale drug searches are among the highlights of MDA’s research program that viewers will learn about on MDA’s 42nd annual Jerry Lewis Labor Day Telethon, broadcast nationwide Sept. 2-3 via some 190 “Love Network” stations. The show also can be seen on the Internet at www.mda.org.

Comedy legend Jerry Lewis once again will host the show, with help from many entertainment luminaries. In addition to the star-studded line-up, the 21 1/2 -hour show will feature research news and profiles of people who have one of the more than 40 neuromuscular diseases in MDA’s program.

“We’re in the age of trials – and that’s a good thing!” enthused Lewis, 81. “Research is moving from lab mice to actual tests in humans and production of some experimental medications. This is exciting stuff.”

Among the more than 40 conditions covered by MDA are nine forms of muscular dystrophy, amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease), myasthenia gravis, spinal muscular atrophy (SMA) and Charcot-Marie-Tooth disease (CMT).

The 2006 Telethon was viewed by more than 40 million people, and its final tote board figure was a record $61 million.

In addition to research, Telethon funds provide direct medical care through the Association’s 225 clinics and 37 MDA/ALS centers; recreation for children with neuromuscular disease at accessible summer camps around the United States; durable medical equipment, including communication devices; more than 300 support groups; and a comprehensive educational program.

MDA dedicates 77.4 cents of every dollar it spends to services.

Research Progress

This year, MDA has allocated $33.6 million for research, funding some 350 projects worldwide. MDA-funded scientists are making progress in decoding disease mechanisms and identifying and testing promising treatments.

• In November 2006, MDA, with the Phoenix-based Translational Genomics Research Institute (TGen), announced the identification of several potentially significant genetic differences in people who develop sporadic (non-inherited) ALS. Further analysis is under way.
• In January of this year, the Association, in collaboration with the ALS Therapy Development Institute (ALS-TDI) in Cambridge, Mass., launched a $36 million effort to identify biochemical targets in ALS and find drugs that interact with them.
• At the same time, MDA joined other muscular dystrophy groups from around the world in forming the Duchenne Research Collaborative International, a coalition to pool knowledge and resources and accelerate treatment development in Duchenne muscular dystrophy (DMD).

Since last year’s Telethon, researchers also have:

• continued with an MDA-funded gene therapy trial in DMD, in which a functional version of the dystrophin gene was injected into an arm muscle in six DMD-affected boys;
• witnessed continued, higher-dose human testing of PTC124, a drug developed by a biotech company with help from MDA, that allows cells to “read through” a molecular stop signal present in some forms of DMD, and produce the functional dystrophin protein that’s lacking in the disease;
• witnessed the launch of the first human trial of “exon skipping,” a strategy that may allow DMD-affected muscle cells to skip over a variety of genetic errors, after MDA-supported research helped develop this approach at the laboratory stage;
• developed some 300 “exon skipping” compounds that may make it possible for muscle cells to skip over a wide range of genetic errors in the gene for dystrophin;
• helped patients with Pompe’s disease (acid maltase deficiency) receive the recently approved drug Myozyme, an enzyme replacement therapy that grew out of MDA-supported scientific research and developed into a medication by the pharmaceutical company Genzyme;
• identified a molecular “brake” that keeps the potentially therapeutic protein utrophin from spreading throughout muscle fibers and compensating for the missing dystrophin protein in DMD;
• identified a compound called trichostatin that allows mice with SMA to produce more full-length SMN protein, a deficiency of which causes this disease;
• found that nervous system cells located near cells directly affected by ALS secrete a toxic compound that kills these cells in at least one form of the disease, opening up new avenues for investigative treatments;
• blocked a pathway that controls inflammation in mice with DMD and found that the mice regenerated some of their muscle tissue and were able to breathe better than untreated mice;
• identified a new type of muscle stem cell that, when injected into mice with DMD, produced mature muscle fibers and improved the animals’ muscle function; and
• tested two drugs that prevent excess deposition of damaging scar tissue in muscle in mice with DMD, opening new possibilities for treatment.

Comprehensive Services

MDA is allocating approximately $68.5 million this year to health care services for people with neuromuscular diseases. With help from Telethon donations, the Association:

• registers tens of thousands of visitors to 225 hospital-affiliated clinics and 37 MDA/ALS centers, for diagnosis, follow-up treatment, and physical, occupational and respiratory therapy consultations;
• offers financial assistance with the purchase or repair of wheelchairs, leg braces and communication devices;
• lends thousands of pieces of adaptive equipment, such as hydraulic lifts, wheelchairs and hospital beds;
• sends more than 4,000 kids to accessible MDA summer camps at no cost to their families;
• pays for flu vaccinations to help prevent potentially deadly respiratory complications of neuromuscular disease; and
• facilitates more than 300 support groups, where those affected by a neuromuscular disease and their families and caregivers can socialize and exchange information.

Professional and Public Health Education

Telethon donations help MDA fund scientific and medical conferences among researchers, physicians and other health care professionals.

Contributions also make possible the publication of informative booklets, care guides, videos, the award-winning Quest magazine, monthly MDA/ALS Newsmagazine, and educational booklets for those who have just received a diagnosis of a neuromuscular disease.

MDA shares its message through public speaking, media interviews, educational videos and four Web sites, including one in Spanish (www.mda.org, www.mdaenespanol.org, http://www.als-mda.org and www.augiesquest.org).